The Myelin Project Releases RSS Feed

Sisters rally genetic researchers to tackle fatal brain disease

noemail@myelin.org — Fri, 06 Nov 2009 17:00:00 GMT

Tom Avril Philadelphia Inquirer (PA) - Nov. 06, 2009 Nov. 6--The busy scientist was about to leave a conference in Washington for his native Hungary when he was buttonholed by a persistent Main Line mother worried about her son's health. Amber Salzman told him he absolutely had to meet a researcher in France who was working on the deadly brain disease that could strike her son. He said, sure, someday. But after boarding the plane to Hungary, he got a surprise message from the flight attendant: Salzman had already set up the meeting, and the French researcher would meet him in Budapest. That fast-track meeting, and plenty of others arranged by Salzman and her sister Rachel, eventually led to pioneering research published today in the journal Science. The authors report that a novel gene therapy seems to stop progression of ALD, the rare disease featured in the movie Lorenzo's Oil. The study describes results from just two patients, and the therapy is...

Gene therapy technique slows brain disease

noemail@myelin.org — Thu, 05 Nov 2009 20:00:00 GMT

A strategy that combines gene therapy with blood stem cell therapy may be a useful tool for treating a fatal brain disease, French researchers have found. These findings appear in the 6 November 2009 issue of the journal Science, which is published by AAAS, the nonprofit science society. In a pilot study of two patients monitored for two years, an international team of researchers slowed the onset of the debilitating brain disease X-linked adrenoleukodystrophy (ALD) using a lentiviral vector to introduce a therapeutic gene into patient's blood cells. Although studies with larger cohorts of patients are needed, these results suggest that gene therapy with lentiviral vectors, which are derived from disabled versions of human immunodeficiency virus (HIV), could potentially become instrumental in treating a broad range of human disorders. This is the first time we were able to successfully use an HIV-derived lentivirus vector for gene therapy in humans, and also the first time that a...

Gene Therapy Advance Saves Two Boys From Rare Brain Disease

noemail@myelin.org — Thu, 05 Nov 2009 19:00:00 GMT

Nov. 5 (Bloomberg) -- Two seven-year-old boys with a fatal brain disease who couldn’t get bone marrow transplants were saved by scientists whose gene therapy technique may let doctors treat other incurable disorders. Doctors in Paris delivered the gene into the boys’ bodies using HIV, the virus that causes AIDS. The virus, stripped of genetic material that makes it toxic, integrates permanently into the DNA of cells it enters, scientists said. That means the modified gene remains in the blood-forming stem cells for the life of the patient, according to a report in the journal Science. Two years after the experimental treatment, the neural damage has been halted or reversed and the two boys attend school and lead normal lives, said Nathalie Cartier, the study’s lead author. The treatment was cited as an example of a comeback for gene therapy, after years of setbacks, in an editorial in the journal. Their disease is completely stabilized, they are fine and there’s no reason this...

Brain Disease Treated by Gene Therapy

noemail@myelin.org — Thu, 05 Nov 2009 19:00:00 GMT

A treatment based on HIV finds first success in humans. The successful treatment of a brain disease marks a big step forward for gene therapy.Getty Researchers have halted a fatal brain disease by delivering a therapeutic gene to the stem cells that mature into blood cells. The gene was transferred using a virus derived from HIV, a technique that researchers have pursued for more than a decade but has not been successful in humans until now. Together with his colleagues, paediatric neurologist Patrick Aubourg at INSERM — France's main biomedical research agency — and at the Saint-Vincent de Paul Hospital in Paris, developed the system to treat X-linked adrenoleukodystrophy (ALD), a neurodegenerative disease that affects young males. ALD results in severe degeneration of the myelin sheath, a structure that is crucial for brain-cell function. The disease is caused by mutations in a gene encoding the ALD protein, which has an important role in cells that make up the myelin sheath...

Scientists Halt Brain Disease with New Gene Therapy

noemail@myelin.org — Thu, 05 Nov 2009 19:00:00 GMT

LONDON (Reuters) - Scientists have managed to halt a rare and fatal brain disease with an experimental gene therapy technique using a deactivated version of the AIDS virus, a study published on Thursday showed. The international team used a disabled form of human immunodeficiency virus (HIV) to deliver working genes to two boys with the brain disease X-linked adrenoleukodystrophy (ALD). Their success may help shape future treatment. Patrick Aubourg of Inserm-University Paris Descartes, who led the study, said it was the first time scientists had successfully used an HIV-derived delivery technique for gene therapy in humans, and the first time gene therapy had been used effectively in a severe brain disease. Up to now we have treated two boys ... and we can say with confidence that more than two years after gene therapy their brain disease has been arrested, he said. Featured in the 1992 movie Lorenzo's Oil, ALD is a rare hereditary condition caused by a deficiency of a protein...

Duke Studies New Approach in Fetal Transplants for Metabolic Disorders

noemail@myelin.org — Thu, 15 Oct 2009 19:00:00 GMT

October 13th, 2009 --> (PhysOrg.com) -- Researchers say a new development in cord blood transplants for inherited metabolic disorders may be curative for some babies who are treated while still in the womb. Joanne Kurtzberg, MD, professor of pediatrics and pathology and director of the Pediatric Blood and Marrow Transplant Program at Duke University Medical Center, says the new approach uses a small, select number of therapeutic stem cells that have been treated to hasten and improve the process of engraftment. Kurtzberg is formally studying the procedure in a pilot trial open to newly pregnant mothers known to be at risk for having children with lethal metabolic disorders. Metabolic disorders -- also known as lysosomal storage disorders -- include several dozen rare, inherited genetic diseases marked by the absence of specific enzymes the body needs to break down and get rid of metabolic byproducts of energy production. Left untreated, metabolic disorders can lead to bone,...

Pioneering HHS HRSA Grant Funds Establishment of First National Newborn Screening Clearinghouse

noemail@myelin.org — Thu, 24 Sep 2009 18:30:00 GMT

WASHINGTON – September 16, 2009 – The nation’s first Newborn Screening Clearinghouse (NBSC), connecting millions of parents and healthcare providers with resources and information relevant to more than four million newborns screened annually, will be created through a $3.75 million cooperative agreement from the U.S. Health and Human Services, Health Resources and Services Administration (HRSA), Genetic Services Branch. The project will span five years. Genetic Alliance and the National Newborn Screening and Genetics Resource Center (NNSGRC) at the University of Texas Health Science Center at San Antonio, will develop the NBSC with the Genetics and Newborn Screening Regional Collaborative Groups, March of Dimes, the Association of Public Health Laboratories and many other partners. For more than 40 years infants born in the U.S. have been screened for an increasing number of congenital conditions, yet parents are often unaware of the number and quality of screening their children...

Opexa Reports Additional Favorable Data with Tovaxin for Multiple Sclerosis

noemail@myelin.org — Thu, 24 Sep 2009 18:00:00 GMT

Opexa Reports Additional Favorable Data with Tovaxin® for Multiple Sclerosis THE WOODLANDS, Texas (September 8, 2009) – Opexa Therapeutics, Inc. (NASDAQ: OPXA), a company developing Tovaxin®, a personalized T-cell immunotherapy for multiple sclerosis (MS), today announced results from further analysis of the double-blind, placebo-controlled, 52-week Phase IIb TERMS clinical study of 150 patients with Relapsing Remitting MS (RRMS). This analysis evaluated patients with an annualized relapse rate of one or greater at study entry (ARR≥1). More than 83% of the Tovaxin-treated group (n=85) remained relapse free at one year and the annualized relapse rate after treatment decreased to 0.20, a 42% reduction compared to placebo. The results of this expanded analysis confirm those found in the previously-reported per-protocol analysis of patients in the TERMS study with ARR>1. This post-hoc analysis which represents 86% of the total patient population in the TERMS study...

Opexa Announces Stem Cell Agreement with Leading

noemail@myelin.org — Thu, 10 Sep 2009 16:00:00 GMT

Opexa Announces Stem Cell Agreement with Leading Global Pharmaceutical Company THE WOODLANDS, Texas (August 7, 2009) – Opexa Therapeutics, Inc. (NASDAQ: OPXA), a company developing a novel T-cell immunotherapy for multiple sclerosis (MS), today announced that it has entered into an exclusive agreement with Novartis, one of the world’s largest pharmaceutical companies, for the further development of Opexa’s novel stem cell technology. This technology, which has generated preliminary data showing the potential to generate monocyte derived islet cells from peripheral blood mononuclear cells, was in early preclinical development at Opexa. Under the terms of the agreement, Novartis will acquire the stem cell technology from Opexa and Novartis will have full responsibility for funding and carrying out all research, development and commercialization activities. Opexa will receive an upfront cash payment of $3 million, plus an additional $1 million as a technology transfer fee to be...

American Brain Coalition Newsletter Highlights The Myelin Project

noemail@myelin.org — Fri, 17 Jul 2009 14:00:00 GMT

/attachments/wysiwyg/1/AmericanBrainCoalition.htm

Genetic Alliance Issues Statement Applauding New NIH Therapeutics for Rare and Neglected Diseases Program

noemail@myelin.org — Thu, 21 May 2009 19:00:00 GMT

WASHINGTON – May 20, 2009 – Earlier today, the National Institutes of Health (NIH) announced the establishment of a new initiative called the Therapeutics for Rare and Neglected Diseases (TRND) Program, a federally mandated effort to encourage and speed the development of new drugs for rare and neglected diseases. Sharon Terry, president and chief executive officer of Genetic Alliance, a nonprofit health advocacy organization, issued the following comments in response to the establishment of TRND: Today marks a leap forward in transforming the rare and neglected disease drug development pipeline. NIH is a crucial entity working in this space. In the United States, a rare disease is defined as a condition affecting fewer than 200,000 people, and diseases lacking substantial therapeutic development activity are considered neglected diseases. Many genetic diseases are both rare and neglected, and collectively there are more than 6,000 rare and neglected diseases affecting over 25...

NORTHWOOD SCHOOL STUDENTS RAISE MONEY FOR MYELIN DISEASE RESEARCH

noemail@myelin.org — Thu, 14 May 2009 15:00:00 GMT

The Myelin Project Each year the student body raises money for The Myelin Project which was established in 1989 with the aim of funding research to find a cure for demyelinating diseases, such as the leukodystrophies which are genetic and multiple sclerosis which is acquired. In 1997, when Tommy Fleming, son of Tom and school nurse Karen, was diagnosed with MS, the most common Myelin disorder, the school planned to have an annual fundraiser. This year, the rains thwarted many of our planned fundraising activities, but the students were not to be daunted by the weather! After a successful dance held the night before, the enthusiasm continued the next morning as a hearty group of about 45 students walked around the lake in unison, splashing in puddles and wearing their handmade tie-dyed Myelin t-shirts in support of this cause. The bake sale was moved inside, and many day student families, faculty and students brought in their sweet donations to raise funds. The...

MS NEWS - Ex-Salesman Uses Net to Inform, Persuade

noemail@myelin.org — Thu, 07 May 2009 16:00:00 GMT

You may read the article in its entirety here

http://www.nationalmssociety.org/online-community/personal-stories/stuart-schlossman/index.aspx

Augusto Odone New Investigator Award for AMN research

noemail@myelin.org — Thu, 16 Apr 2009 20:00:00 GMT

TREATING THE OTHER FACE OF X-ALD It is a great pleasure to announce the launch of the Augusto Odone New Investigator Award, a project co-funded by The Myelin Project and Olivers Army http://www.oliversarmy.org Young scientists embarking on their research career are invited to submit their plans for a project aimed at finding treatments for adrenomyeloneuropathy (AMN). This debilitating disorder and X-linked adrenoleukodystrophy (X-ALD) are caused by a mutation in the same gene (known as the ALD gene). While X-ALD has been the focus of much research, very little is known about AMN. This new award is intended to help bridge the gap. Most of the visitors to this website know something about X-ALD. This genetic disease affects about one-third of boys who carry the X-ALD gene, causing loss of most normal neurologic functions within one or two years. Fewer than 20 years ago, X-ALD was invariably fatal. Thanks, in part, to The Myelin Project, substantial progress has been made in...

Hope for the Future: Embryonic Stem Cells

noemail@myelin.org — Tue, 31 Mar 2009 23:00:00 GMT

The American Brain Coalition (ABC) is a non-profit organization that brings together people with disabling brain disorders, the families of those that are affected, and the professionals that research and treat diseases of the brain. The mission of the ABC is to reduce the burden of brain disorders, and advance the understanding of the brain. Stem Cell Research Hope for the Future: Embryonic Stem Cells ABC along with over 70 percent of the American public, supports federal funding for embryonic stem cell research. This research holds much promise in better understanding brain disorders and for developing new treatments and possibly cures for people with brain disorders. Embryonic stem cells hold immense promise for those suffering from neurological diseases, such as Parkinson’s, and spinal cord injury. These diseases, and many others, are caused by damage to a patient’s cells and tissue. Stem cells can genetically mirror the healthy cells of a living person. In other words,...

Developing Treatments for Adrenomyeloneuropathy and Multiple Sclerosis

noemail@myelin.org — Fri, 27 Mar 2009 20:00:00 GMT

Financial News Nutra Pharma Corp. (NPHC.OB) Subsidiary Introduces Cost Effective Contract Research Services Scottsdale, Arizona 3/27/2009 06:17 PM GMT (TransWorldNews) QualityStocks would like to highlight Nutra Pharma Corp. (OTCBB: NPHC). Nutra Pharma is a biopharmaceutical company specializing in the acquisition, licensing and commercialization of pharmaceutical products and technologies for the management of neurological disorders, cancer, autoimmune and infectious diseases. The company, through its subsidiaries, carries out basic drug discovery research and clinical development and also seeks strategic licensing partnerships to reduce the risks associated with the drug development process. In the company’s news yesterday, Nutra Pharma Corp., a biotechnology company focused on developing treatments for Adrenomyeloneuropathy (AMN), HIV and Multiple Sclerosis (MS), announced that its wholly-owned drug discovery subsidiary, ReceptoPharm, has introduced a line of...

New Research Award for AMN

noemail@myelin.org — Wed, 18 Mar 2009 13:00:00 GMT

The Myelin Project announces the creation of the Augusto Odone Young Investigator Award. This Award is created to encourage promising young investigators to pursue a career in remyelination research. This Research Award will be co-funded by Oliver's Army and The Myelin Project.

This first Award is aimed at research of adrenomyeloneuropathy (AMN).

Adrenomyeloneuropathy (AMN) is a rare inherited metabolic disorder that is a form of Adrenoleukodystrophy (ALD), which occurs in young men. AMN and ALD are two distinct diseases arising from a mutation in the same gene (the ALD gene). Approximately 1/3 of the boys who carry the ALD gene will (if not treated with Lorenzo's oil) develop ALD and die within 1 to 5 years. The remaining 2/3 will develop AMD as young adults. About 1/2 to 2/3 of the mothers will develop AMN in middle age. So, while ALD has received a lot of publicity and been the subject of much rsearch, AMN research has been largely overlooked.

Maximum research award will be $150,000 per year for 2 years. An additional year will be possible with demonstration of satisfactory progress.

Preference will be given to projects that are translational/clinical in nature.

Details for submission will be available on April 1, 2009 on our web site

MYELIN PROJECT OF CANADA: UNA SERATA CONTRO LA SCLEROSI MULTIPLA

noemail@myelin.org — Thu, 12 Mar 2009 23:15:00 GMT

Marilena Carinci, insieme ad un gruppo di amiche e con la fattiva collaborazione dell'ufficio del Consigliere Regionale di Vaughan Gino Rosati, ha organizzato una serata a favore della ricerca sulla Sclerosi Multipla.
Ospite della serata è stato Augusto Odone ed è stato proiettata una clip del film "L'olio di Lorenzo", basato sulla storia vera della ricerca disperata di una medicina per Lorenza deceduto lo scorso anno all'età di 30 anni.
Laura Pauline Mammone, vincitrice del Vaughan Fame Award, si è esibita nel corso della serata con un vasto repertorio canoro.

Massimiliano Galassi

Myelin Board Member Chris Kaag Receives Alumni Achievement Award

noemail@myelin.org — Tue, 17 Feb 2009 22:00:00 GMT

It gives me great pleasure to share with you today the exciting news that Penn State Berks alumnus Christopher Kaag ' 04 has been chosen by University Park as one of ten alumni to receive the 2009 Alumni Achievement Award. The Alumni Achievement Award recognizes alumni 35 years of age and younger for their extraordinary professional accomplishments. Recipients of the award are invited to return to the campus to share their expertise with students, faculty and administrators. They are to demonstrate to students that Penn State alumni succeed in exceptional fashion at an early age. Chris was diagnosed at the age of 21 with a degenerative nerve disorder. Since then he has maintained a positive attitude and stays focused on his goals. He has combined his passion for entrepreneurialship, fitness and charity into a successful business, charitable organization and annual triathlon. In 2004, Chris founded, Got the Nerve?, a triathlon designed to raise money for the Meylin...

Aldagen Receives Orphan Drug Designation for ALD-101

noemail@myelin.org — Thu, 15 Jan 2009 16:00:00 GMT

Aldagen Receives Orphan Drug Designation for ALD-101 ALD-101, a Novel Stem Cell Therapy, Is Currently in Phase 3 Trial DURHAM, NC -- (Marketwire) -- 10/30/08 -- Aldagen, Inc. today announced that it hasreceived orphan drug designation for ALD-101 from the U.S. Food and DrugAdministration (FDA). The designation was granted for the use of ALD-101to improve patient outcomes by decreasing time to platelet and neutrophilengraftment in patients with inherited metabolic disorders undergoingumbilical cord blood transplantation. Aldagen is currently conducting a Phase 3 trial infusing ALD-101 inpatients with inherited metabolic disorders undergoing umbilical cord bloodtransplantation. Cord blood transplantation is commonly used to treatchildren with inherited metabolic diseases, including Krabbe syndrome,metachromatic leukodystrophy, Hurler syndrome and adrenoleukodystrophy.These diseases are progressive, degenerative and often fatal. In manycases, the only treatment available to these...